SparingVision Recruiting for Clinical Trial of Cone-Preserving Gene Therapy for RP

SparingVision, a French company developing therapies for ocular conditions including inherited retinal diseases, has dosed the first cohort of patients at the low dose in its Phase 1/2 clinical trial for its gene-independent, cone-preserving therapy known as SPVN06 for retinitis pigmentosa (RP). The company received a positive recommendation from the Data Safety Monitoring Board (DSMB) to dose the second cohort at the medium dose. Known as PRODYGY, the trial is being conducted at the University of Pittsburgh Medical Center and Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts (CHNO XV-XX), Paris.

SparingVision plans to enroll a total of 33 RP patients who have disease-causing mutations in PDE6A, PDE6B, or RHO. Initially, the trial is enrolling patients with visual acuity between 20/200 and 20/400 and a visual field of 20 degrees or less. Patients with better vision will be enrolled later in the Phase 1/2 trial.

The Foundation’s RD Fund, a venture philanthropy fund for emerging therapies that are in or nearing early-stage clinical trials, is one of the funders for SparingVision. The Foundation also provided several years of research grant funding for the preclinical development of SPVN06.

SPVN06 expresses a protein called rod-derived cone-viability factor (RdCVF), a protein naturally occurring in the retina identified by SparingVision co-founders José Sahel, MD, and Thierry Léveillard, PhD, at the Institut de la Vision. The scientists demonstrated in laboratory studies that RdCVF prevented or slowed the degeneration of cones, the cells in the retina that provide central and color vision and enable people to read, drive, and recognize faces. RdCVF is naturally secreted by rods, the retinal cells that provide night and peripheral vision. In people with RP, the progressive loss of rods leads to loss of cones.

SPVN06 is administrated through a one-time subretinal injection. The therapy uses a human-engineered adeno-associated virus (AAV), which works like a vast container system, to deliver DNA which expresses RdCVF in retinal cells.

RP affects more than two million people worldwide. The retinal disease is usually diagnosed in childhood, progressively leading to legal or total blindness in adulthood.

Further details can be found on Clinicaltrials.gov (https://clinicaltrials.gov/study/NCT05748873) or by contacting the genetic counselors at University of Pittsburgh Medical Center at: retinal.dystrophy@upmc.edu.